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CStone receives approval in China to initiate Phase 1 clinical trial for RET inhibitor BLU-667 (CS3009)

On March 21, 2019 CStone Pharmaceuticals ("CStone"; HKEX: 2616) reported that China’s National Medical Products Administration (NMPA) has approved the clinical trial application to begin a Phase 1 trial in China for BLU-667 (CS3009), a highly selective and potent RET inhibitor discovered by CStone’s partner Blueprint Medicines (Press release, CStone Pharmaceauticals, MAR 21, 2019, View Source [SID1234534561]). The study is part of Blueprint Medicines’ ongoing, global Phase 1 ARROW clinical trial for patients with RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other advanced solid tumors. Trial objectives include overall clinical response, duration of response, pharmacokinetics, pharmacodynamics and safety.

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In June 2018, CStone and Blueprint Medicines entered into a license and collaboration agreement in which Blueprint Medicines granted CStone exclusive rights to develop and commercialize BLU-667 and two other drug candidates in Mainland China, Hong Kong, Macau and Taiwan.

Recently, BLU-667 was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of RET-mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments.

Based on the early clinical data and regulatory feedback, Blueprint Medicines has announced plans to submit a New Drug Application (NDA) to the U.S. FDA for BLU-667 in the first half of 2020.

"BLU-667 has already demonstrated its potential to produce clinical responses in several RET-altered tumor types, and there are currently no selective RET inhibitors approved globally," noted CStone Chairman and CEO Dr. Frank Jiang. "If the data generated in Chinese patients are consistent with global results, we plan to use the global and China data from the ARROW study to support NDA filings in China."

CStone’s Chief Medical Officer Dr. Jason Yang commented: "Currently available data show that non-small cell lung cancer and medullary thyroid cancer patients with RET altered tumors may benefit from BLU-667, with the potential to advance standards of care in these genetically defined populations. We will move forward on development of BLU-667 in China and hope to make this drug candidate available to Chinese patients as quickly as possible."

About BLU-667

BLU-667 is an investigational, once-daily oral precision therapy specifically designed for highly potent and selective targeting of oncogenic RET alterations. In preclinical studies, BLU-667 consistently demonstrated sub-nanomolar potency against the most common RET fusions, activating mutations and predicted resistance mutations. In addition, BLU-667 demonstrated markedly improved selectivity for RET compared to approved multi-kinase inhibitors, including more than 80-fold improved potency for RET versus VEGFR2. By suppressing primary and secondary mutants, BLU-667 has the potential to overcome and prevent the emergence of clinical resistance. This approach is expected to enable durable clinical responses across the range of RET alterations, with a favorable safety profile.

BLU-667 was designed by Blueprint Medicines’ research team, leveraging the company’s proprietary compound library. Blueprint Medicines is developing BLU-667 for the treatment of people with RET-altered NSCLC, MTC and other solid tumors. The U.S. FDA has granted Breakthrough Therapy Designation to BLU-667 for the treatment of RET-mutation-positive MTC.

Study Finds Cancer Drugs Can be Repurposed Beyond Known Uses

On March 21, 2019 In an article published today by SAGE Publishing, investigators from The Nagourney Cancer Institute and The Albert Einstein Israelite Hospital in São Paulo, Brazil, reported a new approach to identifying effective treatments for patients with advanced metastatic cancers (Press release, Nagourney Cancer Institute, MAR 21, 2019, View Source [SID1234534558]). A drug commonly used to treat kidney and liver cancer was discovered effective for breast cancer, which may open new drug therapies to patients regardless of tumor type. The full article can be found at View Source

The study reports the use of 3-dimensional-human-tumor derived organoids that were isolated directly from a patient’s drug resistant breast cancer. The drug identified, sorafenib (Nexavar), is widely used for the treatment of kidney and liver cancers but has not been found active, used, nor approved for patients with breast cancer.

"The finding of sorafenib activity was unexpected," said Dr. Robert Nagourney, one of the authors of the paper and the director for the laboratory where the studies were conducted.

When the patient with disease that had spread throughout her body had a near complete remission with only two months of oral therapy, a remission that lasted for over a year, the researchers examined possible mechanism for the response and believe that it reflects an entirely new form of cancer cell death known as "ferroptosis." This iron-mediated process of cancer cell death is uniquely activated by sorafenib.

"Of the many targeted drugs that we tested on the cancer cells from a woman who had few options remaining, it was only sorafenib that had this effect, prompting our colleagues in São Paulo to begin therapy immediately," said Dr. Nagourney. "We are fortunate to have fantastic collaborators in Brazil who tested this therapy to such good effect."

The results could not have been more dramatic as the patient showed compete metabolic response by PET/CT only weeks later.

"We are witness to a new concept in cancer medicine where drugs can be identified and selected for individual patients in the laboratory regardless of the tumor type, that is, breast versus kidney versus lung. Drugs do not know what disease for which they were invented," he added.

The drug selection technique employed by Dr. Nagourney was developed by scientists in his laboratory in California. It uses fresh biopsies removed directly from patients to study each cancer patient’s biology to select from amongst dozens of drugs and combinations, those that will work the best. This is but one more example of what is known as repurposing drugs, allowing physicians to outsmart cancers by going around their defenses.

"It is the availability of the EVA/PCD testing platform that for this first time is making it possible to make rational drug choices for cancer patients based upon each individual’s unique features," Nagourney concluded.

EDAP TMS SA : EDAP TMS SA to Announce Fourth Quarter and Fiscal Year 2018 Financial Results on Monday, April 1, 2019

On March 21, 2019 EDAP TMS SA (Nasdaq: EDAP), the global leader in therapeutic ultrasound, reported that it will release its financial results for the fourth quarter and year ended December 31, 2018 after the financial markets close on Monday, April 1, 2019 (Press release, EDAP TMS, MAR 21, 2019, View Source [SID1234534555]).

An accompanying conference call and webcast will be conducted by Philippe Chauveau, Chairman of the Board, Marc Oczachowski, Chief Executive Officer; and François Dietsch, Chief Financial Officer, to review the results. The call will be held at 8:30am EDT on Tuesday, April 2, 2019. Please refer to the information below for conference call dial-in information and webcast registration.

Conference Call & Webcast
Tuesday, April 2nd @ 8:30am Eastern Time
Domestic: 877-451-6152
International: 201-389-0879
Passcode: 13687660
Webcast: View Source

Galera Therapeutics Announces Presentation of Data from Phase 2b Clinical Trial of Avasopasem Manganese at 2019 NCCN Annual Conference

On March 21, 2019 Galera Therapeutics, Inc., a clinical-stage biotechnology company focused on the development of drugs targeting oxygen metabolic pathways with the potential to transform cancer radiotherapy, reported data from its Phase 2b clinical trial of lead product candidate avasopasem manganese for the treatment of severe oral mucositis (SOM) in patients with head and neck cancer will be presented at the 2019 National Comprehensive Cancer Network (NCCN) Annual Conference (Press release, Galera Therapeutics, MAR 21, 2019, View Source [SID1234534553]). The meeting will take place March 21-23, 2019, at the Rosen Shingle Creek in Orlando, Fla.

Details of the presentation are as follows:

Title: Reducing the duration, incidence and severity of mucosal injury due to cancer radiation therapy (RT); positive randomized Phase 2b trial results with GC4419 (avasopasem manganese), a small molecule superoxide (SO) dismutase (SOD) mimetic

Session: General Poster Session Two
Date/Time: Friday, March 22, 2019, 12:25-1:40 p.m. EDT
Presenter: Jon T. Holmlund, M.D., Chief Medical Officer, Galera

As one of the top five scoring abstracts, the data will also be shared in an oral presentation during a breakfast session from 7-8 a.m. EDT on Saturday, March 23, 2019, and published in the Journal of the National Comprehensive Cancer Network print and online editions.

The 2019 NCCN Annual Conference: Improving the Quality, Effectiveness, and Efficiency of Cancer Care, brings together cancer researchers, providers and other care professionals to share the latest cancer therapies and provide updates on selected NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines), the data upon which the NCCN Guidelines are based, and quality initiatives in oncology. For more information, visit View Source

About Avasopasem Manganese

Avasopasem manganese (GC4419) is a highly selective and potent small molecule dismutase mimetic that closely mimics the activity of human superoxide dismutase enzymes. It works to reduce elevated levels of superoxide caused by radiation therapy by rapidly converting superoxide to hydrogen peroxide and oxygen. Left untreated, elevated superoxide can damage noncancerous tissues and lead to debilitating side effects, including oral mucositis (OM), which can limit the anti-tumor efficacy of radiation therapy. Conversion of elevated superoxide to hydrogen peroxide, which is selectively more toxic to cancer cells, can also enhance the effect of radiation on tumors, particularly with stereotactic body radiation therapy (SBRT), which produces high levels of superoxide.

Avasopasem manganese is being studied in the Phase 3 ROMAN trial of patients with head and neck cancer, its lead indication, for its ability to reduce the incidence and severity of radiation-induced severe oral mucositis. In Galera’s 223-patient, double blind, randomized, placebo-controlled Phase 2b clinical trial, avasopasem manganese demonstrated the ability to dramatically reduce the duration of SOM from 19 days to 1.5 days (92 percent), the incidence of SOM through completion of radiation by 34 percent and the severity of patients’ OM by 47 percent, while demonstrating acceptable safety when added to a standard radiotherapy regimen. Avasopasem manganese is also currently being studied in combination with SBRT for its anti-tumor effect in a Phase 1/2 trial of patients with locally advanced pancreatic cancer. In addition, in multiple preclinical studies, it demonstrated an increased tumor response to radiation therapy while preventing toxicity in normal tissue.

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy and Fast Track designations to avasopasem manganese for the reduction of SOM in patients with head and neck cancer.

2018 Annual Report and Notice of Annual General Meeting

On March 21, 2019 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported that its 2018 Annual Report together with the Notice of Annual General Meeting and the Form of Proxy have been posted to shareholders (Press release, Hutchison China MediTech, MAR 21, 2019, View Source [SID1234534549]). The documents can be accessed from the website of Chi-Med (www.chi-med.com).